This study is intended to determine the clinical benefit of tabelecleucel (EBV-specific cytotoxic T-lymphocytes) in subjects with EBV-associated diseases.

Eric Homan - homa0030@umn.edu

Evaluate the efficacy of treatment with bb1111 (also known as LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD).

Danielle Jin - zhuxx003@umn.edu

This protocol aims to characterize the safety and tolerability of loncastuximab tesirine in combination with gemcitabine, lenalidomide, polatuzumab vedotin, or umbralisib, and to identify the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) for any of the combinations in subjects with relapsed or refractory B-cell Non-Hodgkin Lymphoma. This project aims to address the resistance mechanisms to single agent therapies and enhance efficacy by engaging different targets, in synergistic or additive manner.

Marie Hu - hu000322@umn.edu

The proposed study is an individual three-arm randomized controlled tiled aimed at utilizing state-of-the-art intervention methods to examine whether increasing the quality and the quantity of family meals reduces childhood obesity.

Jerica Berge - jberge@umn.edu

The purpose of this study is to find out if using a computer program (called iDose) to guide infliximab dosing is more effective and safer than using standard infliximab dosing over 52 weeks. All patients in this study will be receiving infliximab as part of their medical care, this study is only looking at two different methods of determining the dose and timing of administration.

Beiqing Wu - wu000948@umn.edu

This project is a data and specimen repository for developmental disorders. Participants provide biological samples and permission to store their health-related data. The purpose is collect and manage these materials for use in biomedical research related to developmental disorders.

Williams Dobyns - wbdobyns@umn.edu

The main purpose of this study is to check how safe the study drug is and how well your body handles taking it. The study will also check: • if the study drug works to improve your kidney function • whether the study drug has an impact on your daily life • the amount of the study drug in your blood over a period of time (called pharmacokinetics)

Anael Kuperwajs Cohen - kuper029@umn.edu

Currently, there are no approved treatment options for pediatric subjects with proteinuric kidney conditions. The study will look at the safety, efficacy, and pharmacokinetic (PK)trial in children ≥1 to <18 years treated for up to 108 weeks with the drug sparsentan.

Anael Kuperwajs Cohen - kuper029@umn.edu

This is a treatment guideline for allogeneic hemapoetic stem cell transplant (HSCT) in patients with primary immune deficiencies. Endpoints include time to engraftment, incidence of graft failure, incidence of chimerism at 100 days, 6 months, and 1 year; incidence of acute GVHD at 100 days and chronic GVHD at 6 months and 1 year; incidence of transplant related mortality at 6 months and incidence of disease free survival and overall survival at 6 months. All endpoints will be abstracted from routine data collected by the BMT Database.

Christen Ebens - ebens012@umn.edu

The primary objective is to estimate overall survival (OS) at 3 years post-transplant for patients who received the radiation free preparative regimen BEAM.

Veronika Bachanova - bach0173@umn.edu

This is a Phase 1/2 multicenter, open-label study to evaluate palbociclib in combination with either irinotecan (IRN) and temozolomide (TMZ) or topotecan (TOPO) and cyclophosphamide (CTX) chemotherapy in children, adolescents and young adults with recurrent or refractory solid tumors. The study consists of a non- randomized Phase 1 portion for recurrent or refractory solid tumors followed by potential non- randomized tumor specific cohort(s) and a randomized, Phase 2 portion for recurrent or refractory EWS.

Allison Fullenkamp - fulle631@umn.edu

This study will compare the effectiveness and durability of intensive behavioral counseling vs. medical management plus low-intensity behavioral counseling on BMI, body fat, cardiometabolic risk factors, and quality of life in adolescents with severe obesity. We hypothesize that Wegovy (semaglutide) plus low-intensity behavioral counseling will elicit superior reductions in BMI (primary efficacy endpoint) and body fat and greater improvements in cardiometabolic risk factors and quality of life compared to intensive behavioral counseling at 56 weeks.

Nina Jacobs - njacobs@umn.edu

In this study we want to find out more about weight loss and how diet and medications can affect weight loss. This study will last for up to 58 weeks. There are two phases to the study: - A weight loss phase with prescribe meals that lasts 6 weeks. - A study medication/placebo phase that lasts up 52 weeks. You will not know if you are receiving the medication or the placebo.

Nina Jacobs - njacobs@umn.edu

To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced intensity conditioning regimen based on targeted-exposure busulfan, fludarabine +/- serotherapy in patients with inherited metabolic disorders and severe osteopetrosis.

Paul Orchard - orcha001@umn.edu

This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH (MPS-IH) undergoing laronidase therapy and a stem cell transplant.

Paul Orchard - orcha001@umn.edu

Humanitarian Use device application for DBS therapy for dystonia

Kelly Ryberg - rybe0010@umn.edu

To evaluate 3 year progression free survival (PFS) rate of high-risk neuroblastoma patients after treatment with a tandem consolidation of Thiotepa/ Cyclophosphamide and PBSC rescue followed by Carboplatin/Etoposide/ Melphalan (CEM) and PBSC rescue, as compared to historical controls of a single CEM consolidation course with PBSC rescue.

Holly Franceen - hfrancee@umn.edu

In this protocol, we will enroll pediatric, adolescent and adult patients diagnosed with adrenoleukodystrophy (ALD). These patients will include probands diagnosed by newborn screening and their relatives subsequently diagnosed, as well other patients who are diagnosed with ALD due to other presenting signs and symptoms and subsequently were confirmed to have ALD. We will ask consenting subjects to provide a medical history (with verification via medical records), to participate in a semi-annual health survey and provide consent to collect biospecimens. The overarching goal of this work is to engage with families affected by ALD and to assemble a resource of clinical, medical, and biological data that will allow of to better understand the natural history of ALD, and how this is affected by newborn screening. The initial focus will be on patients within Minnesota, but participation will be open to any family interested in the study, as this will be web-based. This registry and biobank, together with other research conducted in tandem, will possibly provide information describing the natural history of ALD and outcomes with interventions. It is anticipated that the data collected will further our understanding of the natural history of the disease, basic biology of adrenoleukodystrophy, diagnosis and outcomes. Ultimately, this research may lead to new avenues for early diagnosis and development of safer and more effective therapies for ALD.

Rachael Eye - eye00002@umn.edu

This is a study to assess the safety and efficacy of PR001A, an Aden-associated (AAV9) viral vector to treat neuronopathic Gaucher disease type 2 (GD2) in infants. PRA001A will be administered via suboccipital injection to the cisterna magna during a single neurosurgical session. GD2 is a fatal disease of early infancy that does not have any therapeutic options beyond palliative care. This study will enroll infants 0-24 months of age.

Brenda Diethelm-Okita - dieth001@umn.edu

This is a long-term registry is designed to collect data on hypophosphatasia (HPP) to better understand the condition and learn more about the disease, how patients feel about living with HPP and effect of HPP on the patients wellbeing and health. The study will look at participant’s medical records and health questionnaires about the health status of patients. This study collects observational data from clinical care and does not involve any treatment for HPP or administration of medication for HPP.

Kyriakie Sarafoglou - saraf010@umn.edu

The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.

Colleen Correll - corr0250@umn.edu

A single visit study with muscle and/or skin biopsy / blood draw, performed to determine whether a molecular or cellular defect can be attributed to cells of FSHD muscle. This study is recruiting both individuals with genetically confirmed FSHD as well as unaffected healthy (control) individuals.

Ana Mitanoska - mitan001@umn.edu

This is a prospective, longitudinal cohort study involving data collection regarding performance of children with ALD and typically developing (TD)children on neurocognitive testing and collection of neuroimaging data. The first goal of this study is to understand more about how ALD affects a child’s brain and development in childhood as they take part in their normal medical care and monitoring. This is important to identifying the best ways to detect and treat manifestations of ALD such as cerebral ALD. The second goal is to learn about how ALD affects caregivers, so that clinicians can offer better support to families in the future.

Brain Study - brainstudy@umn.edu

This is a treatment protocol for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. There is no research element except the collection of routine clinical data.

Timothy Krepski - tkrepsk1@fairview.org

The objectives of this study are to assess the following: - Incidence of sustained donor engraftment at day 42 post transplant - Incidence of transplant related mortality (TRM) at day 100 - Overall survival at day 100 and 1 year - Acute GVHD after this second transplant at day 100 and 6 months - Chronic GVHD after this second transplant at day 12 and 24 months

Timothy Krepski - tkrepsk1@fairview.org

This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HSCT. While it will primarily be applied for the treatment of non-malignant diseases (NMD), on occasion it may be used to treat patients with malignant disorders as well.

Teresa Bekkala - tkivist1@fairview.org

The purpose of this study is to study the evolution of early life risk factors that may lead to cancer and other conditions. This is a prospective cohort study of families who reside in Minnesota.

Clemen Wilcox - clemenw@umn.edu

The purpose of the research is to find out if atrasentan delays worsening of kidney function in IgAN, FSGS, and Alport Syndrome.

Anael Kuperwajs Cohen - kuper029@umn.edu

Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

Allison Johnston - joh21779@umn.edu

This study is a prospective, non-randomized, observational multi-center study, utilizing a quality improvement methodology to facilitate systematic care coordination, interstage cardiovascular monitoring, and nutritional monitoring into every day practice. Utilizes a national registry to document the impact of these changes on various care processes and outcomes. The aim of this Phase II project intends to: 1) develop and support a robust national registry to gather clinical care process, outcome and developmental data on infants with HLHS between diagnosis and the first year of life, 2) engage pediatric cardiology and cardiac surgery programs in using the registry, and 3) use data from the registry to support and monitor the implementation of QI strategies to standardize and improve care for these infants.

Brittany Faanes - grego318@umn.edu